Clinical trials: Musculoskeletal Deformity
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Marfan Syndrome
The Oxford Marfan Trial
The primary objective of the trial is to estimate the effects of allocation to irbesartan, or doxycycline, or a combination of both irbesartan and doxycycline, compared with placebo, on measures of elastic function of the aorta…
- Trial name
- NCT01949233
- Testing
- Irbesartan 150-300mg capsules daily for 6 months, Doxycycline 100-200mg capsules daily for 6 months
- Where
- 2 sites in United Kingdom
- Volunteers
- 56 people
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy, Performance
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
The investigator investigated the effect of lower limb flexibility on functional performance of children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03534349
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Gastroschisis
Sutureless vs Sutured Gastroschisis Closure
This study aims to prospectively assess outcomes of sutureless versus sutured gastroschisis closure with a randomized control trial. The parameters of this trial were determined using our retrospective study as pilot data.
- Trial name
- NCT01094587
- Testing
- Gastroschisis closure with suture, Gastroschisis closure without suture
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Stanford University
Post-COVID-19 Condition, Ulcerative Colitis
Emerging Indications for Hyperbaric Oxygen Treatment
This study will evaluate the effectiveness of hyperbaric oxygen therapy (HBOT) on treating emerging indications (i.e., conditions that have shown to potentially benefit from HBOT) using the Multicenter Registry for Hyperbaric…
- Conditions
- Post-COVID-19 Condition, Ulcerative Colitis, Crohn Disease, Calciphylaxis
- Trial name
- NCT07240649
- Testing
- Hyperbaric oxygen treatment (HBOT), 100% Oxygen
- Volunteers
- 100 people
- Sponsor
- Jay C. Buckey Jr.
Muscular Dystrophy, Duchenne
Spironolactone Versus Prednisolone in DMD
This is a randomized, open-label, pilot clinical trial of spironolactone suspension versus oral prednisolone for use in Duchenne muscular dystrophy.
- Trial name
- NCT03777319
- Testing
- Spironolactone, Prednisolone
- Where
- 4 sites in United States
- Volunteers
- 2 people
- Sponsor
- Kevin Flanigan
Cleft Lip and Palate
Liposomal Bupivacaine Use in Alveolar Bone Graft Patients
The goal of this clinical trial is to learn if a pain medication called liposomal bupivacaine (brand name EXPAREL®) with epinephrine will provide better pain control, increased activity, and reduced use of opioids compared with…
- Trial name
- NCT06284434
- Testing
- Liposomal Bupivacaine, Bupivacain, Epinephrine
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Kerry O'Rourke
Hemophilia, Arthropathy of Knee
Evaluation of Static Postural Balance in Children With Hemophilia and Its Relationship With Joint Health
Hemophilia is a rare hematological disorder characterized by a partial or complete deficiency of clotting factor VIII or IX.Bleeding disorders mainly affect the musculoskeletal system.
- Conditions
- Hemophilia, Arthropathy of Knee, Arthropathy of Ankle or Foot
- Trial name
- NCT04406519
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Necmettin Erbakan University
Pes Planus
Sensorimotor Technique on Navicular Drop in School Going Children
The goal of the study is to determine effects of sensorimotor technique (SMT) program with short foot exercises on navicular drop and foot posture in school going children in both male and female students of age 7-12.
- Trial name
- NCT07576504
- Testing
- sensorimotor technique, short foot exercise
- Where
- Pakistan
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Riphah International University
Mucolipidosis Type IV
Mucolipidosis Type IV Natural History Study
The primary objectives of the study are: to describe the characteristics of the current international MLIV population; to define the median age at which patients with MLIV achieve or lose developmental milestones; to define the…
- Trial name
- NCT05782387
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Massachusetts General Hospital
Stroke, Hemiplegia Following Ischemic Stroke
Improving STEP in Stroke Patients
Stroke is the leading cause of acquired disability in adults. Stroke causes death in 10% of patients, disability and functional handicap in 60% of cases.
- Conditions
- Stroke, Hemiplegia Following Ischemic Stroke, Disability Physical, Spasticity as Sequela of Stroke
- Trial name
- I-STEP (NCT07403149)
- Testing
- REMARCHE® shoes
- Volunteers
- 58 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cleft Palate
Repair of Oronasal Fistula Using Interpositional Buccal Pad of Fat as a Free Graft in a Cleft Palate Patient
36 cleft palate patients had residual oronasal fistula included in this study divided randomly to two groups group I (control group): the fistula was repaired using two layers (oral and nasal layers) group II (study group): The…
- Trial name
- NCT06807450
- Testing
- closure of oro-nasal fistula in cleft palate patients
- Where
- Egypt
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Tanta University
Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1)
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and…
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- NCT06809049
- Testing
- Music and movement education
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- Hanns Lochmuller
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Gastroschisis
Multi-Centre Gastroschisis Interventional Study Across Sub-Saharan Africa
This study is a multi-centre interventional study at seven tertiary paediatric surgery centres in Ghana, Zambia, Malawi and Tanzania aimed at reducing mortality from gastroschisis.
- Trial name
- NCT03724214
- Testing
- Pre-hospital interventional care bundle, In-hospital interventional care bundle
- Where
- 9 sites in Canada, Ghana and 4 more
- Volunteers
- 235 people
- Sponsor
- King's College London
Club Foot
Midfoot Osteotomy Versus 8-Plate Epiphysiodesis for Residual Metatarsal Adductus
Epiphysiodesis
- Trial name
- NCT07466043
- Testing
- Midfoot Osteotomy, 8-Plate Epiphysiodesis
- Volunteers
- 40 people
- Sponsor
- Assiut University
Duchenne Muscular Dystrophy (DMD)
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy
This is an open-label, single-arm, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of RAG-18 in pediatric patients with Duchenne Muscular…
- Trial name
- NCT07282652
- Testing
- RAG-18
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Peking Union Medical College Hospital
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Pectus Excavatum
Multicenter Trial of Magnetic Mini-Mover for Pectus Excavatum
This study is a Phase 3, multicenter, non-randomized study to evaluate the safety and efficacy of the next-generation outpatient Magnetic Mini-Mover Procedure (3MP) in 15 patients aged 8-14 years for the correction of pectus…
- Trial name
- 3MP (NCT01327274)
- Testing
- Magnetic Mini-Mover Procedure (Magnimplant)
- Where
- 3 sites in United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Michael Harrison
Cleft Palate
Optic Nerve Sheath Diameter in Patients Undergoing Palatoplasty
The purpose of this study is to evaluate the change of the optic nerve sheath diameter according to the change of the neck position in pediatric patients undergoing palatoplasty.
- Trial name
- NCT03585608
- Testing
- Neck extension
- Where
- 2 sites in South Korea
- Volunteers
- 30 people
- Sponsor
- Asan Medical Center
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